Real-Life Experience With Subcutaneous Plasma-Derived C1-Inhibitor for Long-Term Prophylaxis in Patients With Hereditary Angioedema: A Case Series.

Abstract

Hereditary angioedema due to C1-inhibitor deficiency (C1-INH-HAE) is characterized by swelling attacks that may be even life-threatening. To reduce the frequency of attacks, some patients need a long-term prophylaxis (LTP). In addition to the intravenous administration, plasma-derived C1-inhibitor (pdC1-INH) has been proved effective also if administered subcutaneously at the dose of 120 IU/kg/week. In this case series, we collected from clinical records data about 5 patients with poorly controlled C1-INH-HAE with the registered LTPs or with difficult venous access, referred to the angioedema center in Milano (Italy), who received it at lower doses, i.e., 42.86–65.22 IU/kg/week. All the patients experienced a reduction in the attack rate, ranging from 29.67% to 96.53% compared with a control period with a different LTP or with no LTP. For one patient, the comparison was made with a period when he received s.c. pdC1-INH 2 (with poor outcomes) instead of 3 times a week, which made the patient experience a decrease in the attack rate from 5.26 to 1.12 attacks/month. Observation periods varied between 2.6 and 47.97 months. Two patients reported adverse events, which were localized at the infusion site and mild in severity. In conclusion, subcutaneous pdC1-INH represents an alternative therapeutic choice according to the physician's judgment for selected patients with HAE poorly controlled with registered LTPs. In patients with difficult venous access, in countries where pdC1-INH is not approved for subcutaneous administration, about half the recommended dose may be beneficial, although suboptimal results may be obtained.